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Related Concept Videos

Gene Therapy00:59

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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Methods of Nuclear Reprogramming01:24

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Nuclear reprogramming is a process of transforming one cell type into an unrelated cell type by epigenetic changes that alter the cell’s original gene expression pattern. Such epigenetic changes force cells to express a different set of genes, which play a significant role in inducing transformation into other cell types. Nuclear reprogramming offers applications in reproductive cloning for livestock propagation and regenerative medicine — developing patient-specific cells for...
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Microorganisms in Medicine and Therapeutics01:29

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Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
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Stem Cell Therapy for Tissue Regeneration01:21

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Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
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The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
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Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
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Cell and gene therapy.

Rajesh C Rao1, David N Zacks

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Summary
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Gene and cell therapies offer new hope for retinal degenerative diseases by replacing faulty genes and promoting cell survival. Strategies include cell transplantation and in vivo reprogramming for vision restoration.

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Area of Science:

  • Ophthalmology
  • Regenerative Medicine
  • Genetics

Background:

  • Retinal degenerative diseases cause significant vision loss.
  • Current therapies have limitations in addressing both cell death and genetic defects.

Purpose of the Study:

  • To explore combined gene and cell therapy strategies for retinal degenerative diseases.
  • To discuss novel approaches for cell replacement and gene repair in the retina.

Main Methods:

  • Transplantation of wild-type cells to replace degenerating cells.
  • Sequential gene and cell therapy administration.
  • In vivo reprogramming of rod photoreceptors to cone photoreceptors.

Main Results:

  • These strategies aim to restore retinal function and prevent further vision loss.
  • Cutting-edge advances in cell and gene therapy are being investigated.

Conclusions:

  • A dual approach of gene therapy and cell therapy holds promise for treating retinal degenerative diseases.
  • Cellular lineage conversion offers a novel therapeutic avenue for vision restoration.